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. 2014 May 7;311(17):1729-30.
doi: 10.1001/jama.2013.285873.

Research into rare diseases of childhood

Mark L Batshaw  1 , Stephen C Groft  2 , Jeffrey P Krischer  3
Affiliations

Research into rare diseases of childhood

Mark L Batshaw et al. JAMA. .
No abstract available

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Conflict of interest statement

Conflict of Interest Disclosures: All authors have completed and submitted the ICJME Form for Disclosure of Potential Conflicts of Interest and none were reported.

References

    1. Orphan Drug Act, Pub L No. 97–414, FDA 21, CFR pt 316 (1991).
    1. Orphan product designations and approvals. US Food and Drug Administration web site http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm. Accessed November 11, 2013.
    1. Thorat C, Xu K, Freeman SN, et al. What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis. Pediatrics 2012;129(3):516–521. . - PubMed
    1. Research, condition, and disease categories categorical spending project listing by category NIH Rare Diseases web site. http://report.nih.gov/categorical_spending_project_listing.aspx?FY=2012&.... Accessed November 27, 2013.
    1. Hanley WB, Linsao LS, Netley C. The efficacy of dietary therapy for phenylketonuria. CMAJ 1971;104(12):1089–1091. . - PMC - PubMed

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